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Author: Jean-Claude Muller, 穆卓Executive Editor at BtoBioInnovation  jcm9144@gmail.com

SPECIAL REPORT

An Approval and an Inventive Step in Alzheimer’s Disease.

On July 6, 2023, the US FDA granted Full Approval for Eisai and Biogen’s LEQUEMBI (lecanemab-irmb), a 100 mg/ml injection for intravenous use, for the treatment of Alzheimer’s disease. LEQUEMBI is the first approved treatment which reduces the rate of disease progression and slows cognitive decline in adults with mild Alzheimer’s disease. The drug had received accelerated approval pathway on January 6, 2023. LEQEMBI is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril) and insoluble forms of amyloid beta (Aβ).“Today, the FDA approved LEQEMBI under the traditional approval pathway, making LEQEMBI the first and only approved anti-amyloid Alzheimer's disease treatment shown to reduce the rate of disease progression and to slow cognitive impairment in the early and mild dementia stages of the disease. As a research and development-focused company based on our hhc (human health care) concept, we are proud that the results of Eisai's AD research over the past 40 years have been recognized and delivered to people living with this disease in the United States,” said Haruo Naito, Chief Executive Officer at Eisai. “Today marks a breakthrough in the treatment of Alzheimer’s disease, and we are proud to be at the forefront of ushering in a new era of advances for a disease that was previously considered untreatable. We would like to express our sincere appreciation to those who have worked tirelessly to find a treatment for this unrelenting disease, without whom this progress would not be possible,” said Christopher A. Viehbacher, President and Chief Executive Officer of Biogen.

The approval is also expected to trigger Medicare coverage for adults over 65 who have been unable to afford the $26,500-a-year drug. More than 6 million Americans live with Alzheimer's disease.

Not surprisingly the FDA approval includes a box label (black box) warning about the drug's known side effects, which include brain swelling and tiny bleeds. These side effects, called ARIA (amyloid-related imaging abnormalities), occur in some patients who take LEQUEMBI. While most patients won't notice symptoms that typically surface early during treatment, some experience headaches or infusion-related reactions such as fever and chills. The safety language warns "serious and life-threatening events rarely can occur," including strokes and reminds that patients who inherited two copies of the Alzheimer's risk gene ApoE4 are at greater risk of ARIA. About 15% of patients who take the medication have two copies of this susceptibility gene.

In previous reports we had already indicated that, from the point of view of a patient, LEQUEMBI is far from being a panacea.

In the June MedNous issue, Michael Valenzuela, from the University of Sydney reports of “A cell therapy approach to Alzheimer’s disease”.  The article describes an autologous cell therapy comprised of neuroprecursors derived from human hair follicle targeted at the hippocampus, the memory centre of the brain.  The author discovered “that a rare unipotent cell within the human hair follicle was exceptionally neurogenic, meaning that cells naturally and exclusively develop into brain cells. More specifically, the adult human hair follicle contains both unipotent and multipotent stem cells.  According to the author, Alzheimer’s disease progresses to dementia when at least 10 million neurons have been lost in the hippocampus. Replacing these lost neurons with autologous unipotent hair follicle cells may constitute an innovative approach to treat Alzheimer’s patients.

We wish all our readers a nice summer break

Paris, July 7, 2023

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